STAT+: Sarepta reports second patient death after treatment with Duchenne gene therapy
Sarepta said it was halting shipments of its Duchenne gene therapy for patients who can no longer walk, following the death of a second recipient.
Sarepta Therapeutics said Sunday that it was halting shipments of its Duchenne muscular dystrophy gene therapy for patients who can no longer walk, following the death of a second person who received the treatment.
Sarepta disclosed the first patient death — a 16-year-old boy — in March. Both occurred from acute liver failure, a side effect that has been seen with other gene therapies. The company said both patients were non-ambulatory, meaning their disease had progressed to the point they relied on a wheelchair. Most children with Duchenne lose the ability to walk by adolescence.
The company said early Sunday it was working with experts to come up with an enhanced immunosuppressive regimen that could make the therapy, called Elevidys, safer for non-ambulatory patients. It said it would talk with the Food and Drug Administration about the the proposed regimen.
